Vutrisiran vs Patisiran for Amyloidosis

Recruiting in Palo Alto (17 mi)

+57 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Alnylam Pharmaceuticals

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?This trial is testing vutrisiran, an under-the-skin injection, in patients with a genetic condition called hATTR amyloidosis. The medication aims to lower harmful protein levels in their bodies. Vutrisiran is administered as a periodic subcutaneous injection.

Eligibility Criteria

This trial is for adults aged 18-85 with hereditary transthyretin amyloidosis, a condition where abnormal proteins build up in the body. Participants should have specific levels of neurologic impairment and physical functioning, and not have received other TTR-lowering treatments or had a liver transplant.

Inclusion Criteria

I am mostly able to care for myself.

I have hATTR amyloidosis with a TTR mutation.

My nerve function score is within acceptable limits.

+2 more

Exclusion Criteria

You have important liver function test abnormalities.

My heart condition significantly limits my physical activity.

I have received treatment to lower TTR levels before.

+5 more

Participant Groups

The study compares Vutrisiran, given as an injection under the skin every three months, to Patisiran, administered intravenously every three weeks. After 18 months, participants may receive Vutrisiran less frequently in an extension period.

2Treatment groups

Experimental Treatment

Active Control

Group I: Vutrisiran + Vutrisiran (HELIOS-A)Experimental Treatment1 Intervention

Participants will receive vutrisiran 25 mg subcutaneous (SC) injection once every 3 months (q3M) for 18 months during the Treatment Period followed by vutrisiran 50 mg SC injection once every 6 months (q6M) or vutrisiran 25 mg q3M during the Randomized Treatment Extension (RTE) Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing.

Group II: Patisiran + Vutrisiran (HELIOS-A)Active Control2 Interventions

Participants will receive patisiran 0.3 mg/kg intravenous (IV) infusion once every 3 weeks (q3w) for 18 months during the Treatment Period followed by vutrisiran 50 mg SC injection once q6M or vutrisiran 25 mg q3M during the RTE Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing.